RedHill presents positive Phase 2 results for oral opaganib in patients with COVID-19 pneumonia

RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, today announced presentation of the positive Phase 2 safety and efficacy data for oral opaganib (Yeliva^®, ABC294640) in hospitalized patients with COVID-19 pneumonia at the World Microbe Forum (WMF) 2021 (poster #: 5574).

Results and post hoc analyses of data from the 40-patient U.S. Phase 2 study were presented in a poster entitled, "Opaganib, an Oral Sphingosine Kinase-2 (SK2) Inhibitor in COVID-19 Pneumonia: A Randomized, Double-blind, Placebo-controlled Phase 2A Study, in Adult Subjects Hospitalized with SARS-CoV-2 Positive Pneumonia (NCT: 04414618)" . Patients in the study were randomized to receive either opaganib or placebo in addition to standard of care (SoC), predominantly including dexamethasone and/or remdesivir. Findings include:

• 50% of patients treated with opaganib (n=22) reached room air by Day 14 compared to 22% in the placebo group (n=18). The benefit of reaching room air by Day 14 for patients on opaganib was maintained regardless of whether the patients were receiving dexamethasone and/or remdesivir
• 86.4% of patients treated with opaganib were discharged from hospital by Day 14 compared to 55.6% of patients treated with placebo
• Median time to discharge was 6 days for the opaganib group compared to 7.5 days for the placebo group
• 81.8% of opaganib patients achieved a 2-point improvement in the WHO Ordinal Scale compared to 55.6% of patients in the placebo group - achieved in a median time of 6 days versus 7.5 days, respectively
• No significant differences in safety-related measures between the two groups (with diarrhea being the main treatment-emergent difference in tolerability)

The need for an effective oral therapy to treat COVID-19 is clear. Such a therapy would greatly improve our ability to manage this pandemic. These data, from this proof-of-concept clinical study of opaganib in patients with severe COVID-19, suggest a potential role of SK2 inhibition in combating the effects of this virus. With much more data on opaganib expected in the coming weeks, we could make some real progress toward having access to a much-needed oral therapy for patients who currently have a paucity of options available to them."

Kevin Winthrop, MD, MPH, Professor of Infectious Diseases, Oregon Health & Science University, who presented the findings at World Microbe Forum

"Presentation of these positive data from our exploratory Phase 2 study support our growing confidence that opaganib could be the first novel, oral therapy to demonstrate efficacy in the treatment of COVID-19 in a large late-stage study. With the recent completion of enrollment of our 475-patient global Phase 2/3 study, we will have a clearer picture of that in the very near future," said Mark L. Levitt, MD, Ph.D., Medical Director at RedHill. "Opaganib acts on both the cause and effect of COVID-19 via a unique dual antiviral and anti-inflammatory mode of action. Being host-targeted, opaganib is also expected to maintain effect against the emerging SARS-CoV-2 variants, which continue to threaten the progress being made against the pandemic and underscore the urgent need for effective COVID-19 therapeutics."

The global 475-patient Phase 2/3 study of opaganib in severe COVID-19 has been approved in 10 countries and completed enrollment, through 57 participating sites, on June 6th. The primary endpoint of the study is the proportion of patients breathing room air without oxygen support by Day 14.

Additional important outcome measures, such as time to discharge from hospital, improvement according to the World Health Organization Ordinal Scale for Clinical Improvement and incidence of intubation and mortality, will also be captured in the follow-up period of up to 6 weeks. The study received four independent DSMB recommendations to continue following unblinded safety reviews and a futility review. Additionally, an evaluation of the blinded blended intubation and mortality rates to date was encouraging as compared to reported rates of mortality from large platform studies such as RECOVERY, and other studies in similar patient populations.